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儿科异基因造血干细胞移植的新前沿:针对儿童和青少年急性淋巴细胞白血病的新方法。

New frontiers in pediatric Allo-SCT: novel approaches for children and adolescents with ALL.

作者信息

Pulsipher M A, Wayne A S, Schultz K R

机构信息

Division of Hematology and Hematological Malignancies, Primary Children's Hospital, University of Utah School of Medicine/Huntsman Cancer Institute, Salt Lake City, UT, USA.

Division of Hematology, Oncology, and Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, The Norris Comprehensive Cancer Center, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.

出版信息

Bone Marrow Transplant. 2014 Oct;49(10):1259-65. doi: 10.1038/bmt.2014.114. Epub 2014 Jun 16.

DOI:10.1038/bmt.2014.114
PMID:24933210
Abstract

Although most children with ALL can be cured by chemotherapy approaches, allogeneic hematopoietic cell transplant (HCT) therapy offers a better chance of cure to selected high-risk patients in first remission and most children who relapse. Although transplant-related mortality has decreased significantly in the past decade, relapse remains high after HCT for ALL; developing strategies to decrease relapse and improve survival are vital. Recent studies have shown that relapse risk can be accurately defined using measurements of minimal residual disease (MRD) both pre- and post-HCT and by knowing whether patients get GVHD in the first 2 months after transplant. With these risk definitions in hand, investigators are now applying novel agents and immunotherapeutic methods in attempt to lower MRD before transplant and modulate the GVL effect after transplant. With powerful new immunological approaches coming on line, the transplant process itself will likely expand to include pre and/or post-HCT interventions aimed at reducing relapse.

摘要

尽管大多数急性淋巴细胞白血病(ALL)患儿可通过化疗治愈,但异基因造血细胞移植(HCT)疗法为部分首次缓解的高危患者以及大多数复发患儿提供了更好的治愈机会。尽管在过去十年中,移植相关死亡率显著降低,但ALL患者接受HCT后的复发率仍然很高;制定降低复发率和提高生存率的策略至关重要。最近的研究表明,通过测量HCT前后的微小残留病(MRD)以及了解患者在移植后前两个月是否发生移植物抗宿主病(GVHD),可以准确界定复发风险。有了这些风险定义,研究人员现在正在应用新型药物和免疫治疗方法,试图在移植前降低MRD,并在移植后调节移植物抗白血病(GVL)效应。随着强大的新免疫方法不断涌现,移植过程本身可能会扩大,包括旨在减少复发的HCT前和/或HCT后干预措施。

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造血细胞移植治疗血液系统恶性肿瘤的进展。
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Tisagenlecleucel for the treatment of B-cell acute lymphoblastic leukemia.靶向 CD19 的嵌合抗原受体 T 细胞(tisagenlecleucel)治疗 B 细胞急性淋巴细胞白血病。
Expert Rev Anticancer Ther. 2018 Oct;18(10):959-971. doi: 10.1080/14737140.2018.1512411.
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Circulating L-selectin expressing-T cell subsets correlate with the severity of Foxp3 deficiency autoimmune disease.循环中表达L-选择素的T细胞亚群与Foxp3缺陷自身免疫性疾病的严重程度相关。
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Pediatric chronic myeloid leukemia is a unique disease that requires a different approach.小儿慢性粒细胞白血病是一种需要采用不同治疗方法的独特疾病。
Blood. 2016 Jan 28;127(4):392-9. doi: 10.1182/blood-2015-06-648667. Epub 2015 Oct 28.
Intrachromosomal amplification of chromosome 21 is associated with inferior outcomes in children with acute lymphoblastic leukemia treated in contemporary standard-risk children's oncology group studies: a report from the children's oncology group.21 号染色体的染色体内扩增与当代标准风险儿童肿瘤组研究中治疗的儿童急性淋巴细胞白血病患者的不良结局相关:来自儿童肿瘤组的报告。
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